NEWARK, Calif. - CymaBay Therapeutics, Inc. (NASDAQ: NASDAQ:CBAY), a biopharmaceutical company, announced today that the European Medicines Agency (EMA) will review its Marketing Authorization Application (MAA) for the drug seladelpar, designed to treat primary biliary cholangitis (PBC), a chronic liver disease.
This regulatory milestone follows the submission of clinical data from over 500 PBC patients, including results from the Phase 3 RESPONSE study, which indicated that seladelpar significantly improved disease markers and symptoms like itch in PBC patients.
The RESPONSE study, a pivotal trial recently featured in the New England Journal of Medicine, compared the effects of seladelpar to a placebo. It met its primary endpoint, showing statistically significant improvements without a significant increase in adverse events. The MAA also incorporates findings from the ENHANCE study, the long-term ASSURE study, Phase 2 trials, pre-clinical studies, and details on the drug's chemistry and manufacturing processes.
PBC primarily affects women and can lead to cirrhosis and increased liver-related mortality. Symptoms such as itch and fatigue can significantly affect patients' quality of life. The current second-line treatment approved by the EMA does not adequately address these concerns for all patients, highlighting the need for new therapeutic options like seladelpar.
Seladelpar, a first-in-class selective peroxisome proliferator-activated receptor delta (PPARδ) agonist, has been granted Priority Medicines (PRIME) status by the EMA, a designation that accelerates the review of drugs targeting unmet medical needs. The drug is also under priority review by the U.S. Food and Drug Administration (FDA) and has been accepted for review by the U.K.'s Medicines and Healthcare products Regulatory Agency.
CymaBay's Chief Regulatory and Compliance Officer, Klara Dickinson, expressed the company's anticipation for the EMA's review process and the potential approval of seladelpar in Europe. The EMA's Committee for Medicinal Products for Human Use (CHMP) will conduct the review for all 27 European Union member states, as well as Iceland, Liechtenstein, and Norway.
The information in this article is based on a press release statement from CymaBay Therapeutics, Inc.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.